EP2841572A4 - Genetic correction of mutated genes - Google Patents
Genetic correction of mutated genesInfo
- Publication number
- EP2841572A4 EP2841572A4 EP13781472.9A EP13781472A EP2841572A4 EP 2841572 A4 EP2841572 A4 EP 2841572A4 EP 13781472 A EP13781472 A EP 13781472A EP 2841572 A4 EP2841572 A4 EP 2841572A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- mutated genes
- genetic correction
- genetic
- correction
- mutated
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP19179921.2A EP3597741A1 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261639227P | 2012-04-27 | 2012-04-27 | |
PCT/US2013/038536 WO2013163628A2 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP19179921.2A Division EP3597741A1 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
Publications (3)
Publication Number | Publication Date |
---|---|
EP2841572A2 EP2841572A2 (en) | 2015-03-04 |
EP2841572A4 true EP2841572A4 (en) | 2016-03-02 |
EP2841572B1 EP2841572B1 (en) | 2019-06-19 |
Family
ID=49484041
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP13781472.9A Active EP2841572B1 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
EP19179921.2A Pending EP3597741A1 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP19179921.2A Pending EP3597741A1 (en) | 2012-04-27 | 2013-04-26 | Genetic correction of mutated genes |
Country Status (3)
Country | Link |
---|---|
US (2) | US10711256B2 (en) |
EP (2) | EP2841572B1 (en) |
WO (1) | WO2013163628A2 (en) |
Families Citing this family (46)
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US9738879B2 (en) | 2012-04-27 | 2017-08-22 | Duke University | Genetic correction of mutated genes |
WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
MX2015007550A (en) | 2012-12-12 | 2017-02-02 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications. |
US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
EP3004370A4 (en) | 2013-06-05 | 2017-01-11 | Duke University | Rna-guided gene editing and gene regulation |
DK3011032T3 (en) | 2013-06-17 | 2020-01-20 | Broad Inst Inc | MANUFACTURING, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR TARGETING AGAINST AND MODELING DISEASES AND DISORDERS IN POSTMITOTIC CELLS |
DK3011031T3 (en) | 2013-06-17 | 2020-12-21 | Broad Inst Inc | PERFORMANCE AND APPLICATION OF CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR LIVER TARGET DIRECTION AND THERAPY |
SG11201510286QA (en) | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
KR20160089526A (en) | 2013-12-12 | 2016-07-27 | 더 브로드 인스티튜트, 인코퍼레이티드 | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
EP3957735A1 (en) | 2014-03-05 | 2022-02-23 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
DK3116997T3 (en) | 2014-03-10 | 2019-08-19 | Editas Medicine Inc | CRISPR / CAS-RELATED PROCEDURES AND COMPOSITIONS TO TREAT LEVER'S CONGENITAL AMAUROSIS 10 (LCA10) |
US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
EP3116533B1 (en) * | 2014-03-12 | 2020-08-12 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
CA2959130A1 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
WO2016025759A1 (en) | 2014-08-14 | 2016-02-18 | Shen Yuelei | Dna knock-in system |
US11319555B2 (en) | 2014-11-20 | 2022-05-03 | Duke University | Compositions, systems and methods for cell therapy |
WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Protected guide rnas (pgrnas) |
EP3230452A1 (en) | 2014-12-12 | 2017-10-18 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
JP2018522249A (en) | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Evaluation of CAS 9 molecule / guide RNA molecule complex |
EP3289104B1 (en) | 2015-04-29 | 2020-11-04 | New York University | Method for treating high-grade gliomas |
WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
JP6905755B2 (en) | 2015-08-25 | 2021-07-21 | デューク ユニバーシティ | Compositions and Methods to Improve Specificity in Genomic Engineering Using RNA-Guided Endonucleases |
EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
JP7059179B2 (en) | 2015-10-20 | 2022-04-25 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | Methods and products for genetic engineering |
US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
EP3494220A1 (en) | 2016-08-02 | 2019-06-12 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
WO2018144097A1 (en) | 2016-11-04 | 2018-08-09 | Akeagen Llc | Genetically modified non-human animals and methods for producing heavy chain-only antibodies |
WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
EP4252759A3 (en) | 2018-05-11 | 2023-11-08 | Memorial Sloan Kettering Cancer Center | T cell receptors targeting pik3ca mutations and uses thereof |
WO2020093172A1 (en) | 2018-11-08 | 2020-05-14 | Neovasc Tiara Inc. | Ventricular deployment of a transcatheter mitral valve prosthesis |
EP3898958A1 (en) | 2018-12-17 | 2021-10-27 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
WO2021067788A1 (en) | 2019-10-03 | 2021-04-08 | Artisan Development Labs, Inc. | Crispr systems with engineered dual guide nucleic acids |
US11530425B2 (en) | 2019-10-09 | 2022-12-20 | Massachusetts Institute Of Technology | Systems, methods, and compositions for correction of frameshift mutations |
JP2023500884A (en) | 2019-11-06 | 2023-01-11 | アソシエーション・アンスティトゥート・ドゥ・マイオロジー | Combination therapy for muscle disease |
WO2022051418A1 (en) | 2020-09-01 | 2022-03-10 | 64-X, Inc. | Mammalian cells and methods for engineering the same |
WO2022256448A2 (en) | 2021-06-01 | 2022-12-08 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing, or modifying genes |
WO2023167882A1 (en) | 2022-03-01 | 2023-09-07 | Artisan Development Labs, Inc. | Composition and methods for transgene insertion |
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CA2749305A1 (en) * | 2009-01-12 | 2010-07-15 | Jens Boch | Modular dna-binding domains and methods of use |
WO2011036640A2 (en) * | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
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EP2841572A2 (en) | 2015-03-04 |
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US11976307B2 (en) | 2024-05-07 |
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